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Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03893071
Recruitment Status : Recruiting
First Posted : March 28, 2019
Last Update Posted : March 28, 2019
Sponsor:
Information provided by (Responsible Party):
CTD Holdings, Inc.

Tracking Information
First Submitted Date  ICMJE February 20, 2019
First Posted Date  ICMJE March 28, 2019
Last Update Posted Date March 28, 2019
Estimated Study Start Date  ICMJE March 2019
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 26, 2019)
  • Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product [ Time Frame: 1-104 weeks ]
    Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product
  • Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product [ Time Frame: 1-104 weeks ]
    Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product
  • Number of discontinuations due to AEs: Patient Discontinuation, Study Treatment Discontinuation, Study and Site Discontinuation [ Time Frame: 1-104 weeks ]
    Discontinuation, Study and Site Discontinuation
  • Auditory capacity will be measured by behavioral auditory assessment [ Time Frame: 1-104 weeks ]
    Auditory capacity will be measured by behavioral auditory assessment
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: March 26, 2019)
Change from baseline in total score as well as individual National Institute of Health (NIH) Niemann-Pick Disease Type C (NPC) Severity Scale (NCSS) [ Time Frame: 1-104 weeks ]
Change from baseline in total score as well as individual National Institute of Health (NIH) Niemann-Pick Disease Type C (NPC) Severity Scale (NCSS)
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C
Official Title  ICMJE An Open-Label Extension Study of the Long-Term Safety and Efficacy of Intravenous Trappsol® Cyclo (HP-β-CD) in Patients With Niemann-Pick Disease Type C (NPC-1)
Brief Summary The purpose of this study is to provide continued access to treatment for NPC-1 after participation and completion of the Phase I trial CTD-TCNPC-101, when administered at doses of 1500 mg/kg and 2500 mg/kg by slow IV infusion over a period of 8 to 9 hours every two weeks.
Detailed Description

This is an open-label extension study of intravenous HP-β-CD (administered as Trappsol® Cyclo(TM)) in patients with NPC-1 to evaluate long-term safety and efficacy by providing continued access to treatment following completion of study CTD-TCNPC-101. Patients will receive medication at their home or local site under the supervision of a home nurse professional or at the parent site under the supervision of the site principal investigator. Patients will visit the parent site for safety and efficacy evaluations after receiving treatment for 3 months in the extension protocol.

Thereafter, visits to the parent site will be every 6 months until cessation. The study will continue until Trappsol® Cyclo (TM) becomes commercially available or study/site/patient discontinuation. All patients who complete study CTD-TCNCP-101 and pass the screening criteria will be eligible for this study. The study will be directed from parent site in the US. Vital signs, AEs Adverse Events (AEs) and concomitant meds will be recorded by the home nurse professional or deputy at the local site and reported immediately to the parent site for entry into the Electronic Data Capture (EDC) and reporting to the sponsor.

For logistical reasons home infusions will be permitted as long as AEs, infusion details and concomitants medications are reported directly to the parent site by an authorised deputy according to local guidelines. A Safety Review Committee (SRC) consisting of an independent advisor, the medical monitor, and the principal investigator will be established to review AEs and laboratory data throughout the study.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Niemann-Pick Disease, Type C1
Intervention  ICMJE Drug: Hydroxypropyl-β-cyclodextrin
HP-β-CD will be administered as Trappsol® Cyclo 25% (250mg/mL) by slow intravenous infusion over a period of 8 up to 9 hours.
Other Names:
  • Hydroxypropyl-β-cyclodextrin (HP-β-CD)
  • Trappsol® Cyclo
Study Arms  ICMJE Experimental: Hydroxypropyl-β-cyclodextrin IV
Hydroxypropyl-β-cyclodextrin will be administered as Trappsol® Cyclo 25% (250mg/mL) by slow intravenous infusion over a period of 8 up to 9 hours.
Intervention: Drug: Hydroxypropyl-β-cyclodextrin
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: March 26, 2019)
12
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2021
Estimated Primary Completion Date March 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Completion of study CTD-TCNPC-101 with no safety concerns at the final visit
  • Negative urine pregnancy test for females of child bearing potential
  • Patients must be legally resident in the USA with access to healthcare
  • Written, informed consent

Exclusion Criteria:

  • Inability to comply with the proposed protocol assessments or any uncertainty about their ability to give meaningful, informed consent (legal guardian may give consent with subject assent)
  • Concurrent medical conditions representing a contraindication to any of the study medications
  • Grade 3 renal impairment or worse as indicated by estimated Glomerular filtration rate (eGFR) < 60mL/min/1.73m2
  • Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or International Normalised Ratio (INR) >1.8
  • Male patients and female patients of childbearing potential who are not willing to use appropriate birth control (i.e. double barrier birth control) from enrollment until the follow-up visit
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Sharon H Hrynkow, PhD 3869657273 sharon.hrynkow@cyclodex.com
Contact: Karen Rabb +12144763754 Karen.Rabb@synteract.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03893071
Other Study ID Numbers  ICMJE CTD-TCNPC-102
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party CTD Holdings, Inc.
Study Sponsor  ICMJE CTD Holdings, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Caroline Hastings, MD UCSF Benioff Children's Hospital Oakland
PRS Account CTD Holdings, Inc.
Verification Date March 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP